Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 23
- Locations
- 1
- Primary Endpoint
- Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
Study Overview
Brief Summary
The FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis.
It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy).
The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.
Detailed Description
At the end of the study, it will propose to continue the usual quarterly patient follow up, as recommended by the French ALS centers.
Deferiprone can be administered as part of a compassionate use, for patients who want it and who do not have hypoxemia.
We therefore plan a treatment period compassionate relatively short and less than 3 years.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Supportive Care
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 85 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Amyotrophic lateral sclerosis defined in accordance to the El Escorial criteria (possible, probable or defined)
- •18 to 85 years old patient, male or female
- •Patient with social security cover
Exclusion Criteria
- •Achieved respiratory defined by a FVC <70%
- •Evolution of more than 24 months
- •Demented subject
- •Severe malnutrition
- •Patients with treatment potentially at risk of agranulocytosis and neutropenia
- •Patients with a history of agranulocytosis or iatrogenic under haematological disease
- •Incapable of giving consent
- •Indication against MRI
- •Indication against lumbar puncture
- •Patient refused lumbar puncture
- •Hypersensitivity to iron chelators
- •Concomitant treatment with antacids containing aluminum
- •Presence of another serious illness to life-threatening or disabling cons to the use of the treatment mixture of oxygen and nitrous oxide equally
Arms & Interventions
Deferiprone
Deferiprone, 25 to 30 mg/kg per day, oral use
Intervention: Deferiprone (Drug)
Outcomes
Primary Outcomes
Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
Time Frame: V3, V6, V9, V12, V15
Secondary Outcomes
- Comparison of the progression of ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale ) score for 3 months without treatment (V0 to V3) in the period of the first three months under treatment (V3 to V6).(SCREENING, V0, V3, V6)
- The proportion of patients who become non-self-sufficient after 12 months with the appearance of a sub scores ALSFRS-R less than or equal to 2 on swallowing, cut food using utensils or walk.(SCREENING, V0, V3, V6, V9, V12, V15)
- Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g(V3, V6, V9, V12, V15)
- Number of serious and non-serious adverse events(SCREENING, V0, V3, V6, V9, V12, V15)
- Progression of respiratory vital capacity tests(SCREENING, V0, V3, V6, V9, V12, V15)
- Values of R2 * in MRI(V3, V6, V19)
- Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid(V3, V9)
- No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid(V3, V9)